WO2023215863A3 - Rnai agents for modulating snca - Google Patents
Rnai agents for modulating snca Download PDFInfo
- Publication number
- WO2023215863A3 WO2023215863A3 PCT/US2023/066655 US2023066655W WO2023215863A3 WO 2023215863 A3 WO2023215863 A3 WO 2023215863A3 US 2023066655 W US2023066655 W US 2023066655W WO 2023215863 A3 WO2023215863 A3 WO 2023215863A3
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- disease
- snca
- subject
- cell
- amount
- Prior art date
Links
- 108091030071 RNAI Proteins 0.000 title abstract 2
- 101150110423 SNCA gene Proteins 0.000 title 1
- 210000004027 cell Anatomy 0.000 abstract 4
- 102100026882 Alpha-synuclein Human genes 0.000 abstract 3
- 101000834898 Homo sapiens Alpha-synuclein Proteins 0.000 abstract 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 2
- 206010067889 Dementia with Lewy bodies Diseases 0.000 abstract 2
- 208000018737 Parkinson disease Diseases 0.000 abstract 2
- 102000003802 alpha-Synuclein Human genes 0.000 abstract 2
- 108090000185 alpha-Synuclein Proteins 0.000 abstract 2
- 150000001875 compounds Chemical class 0.000 abstract 2
- 230000000694 effects Effects 0.000 abstract 2
- 230000004770 neurodegeneration Effects 0.000 abstract 2
- 208000015122 neurodegenerative disease Diseases 0.000 abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 abstract 2
- 208000024827 Alzheimer disease Diseases 0.000 abstract 1
- 206010012289 Dementia Diseases 0.000 abstract 1
- 208000015872 Gaucher disease Diseases 0.000 abstract 1
- 208000009829 Lewy Body Disease Diseases 0.000 abstract 1
- 201000002832 Lewy body dementia Diseases 0.000 abstract 1
- 208000001089 Multiple system atrophy Diseases 0.000 abstract 1
- 208000009144 Pure autonomic failure Diseases 0.000 abstract 1
- 208000032859 Synucleinopathies Diseases 0.000 abstract 1
- 230000000692 anti-sense effect Effects 0.000 abstract 1
- 230000009368 gene silencing by RNA Effects 0.000 abstract 1
- 208000024891 symptom Diseases 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Public Health (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
Abstract
Provided are antisense agents, pharmaceutical compositions, and methods of use for reducing the amount or activity of alpha-synuclein (SNCA) RNA in a cell or subject, and in certain instances reducing the amount of alpha-synuclein protein in a cell or subject. In certain embodiments, also provided herein are oligomeric compounds, oligomeric duplexes, and RNAi agents for reducing the amount or activity of SNCA RNA in a cell or subject, and in certain instances reducing the amount of SNCA protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such neurodegenerative diseases include synucleinopathies, e.g., Parkinson's disease, dementia with Lewy bodies, diffuse Lewy body disease, Parkinson's disease dementia, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease, and Alzheimer's disease.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202263339292P | 2022-05-06 | 2022-05-06 | |
US63/339,292 | 2022-05-06 |
Publications (2)
Publication Number | Publication Date |
---|---|
WO2023215863A2 WO2023215863A2 (en) | 2023-11-09 |
WO2023215863A3 true WO2023215863A3 (en) | 2023-12-14 |
Family
ID=88647235
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2023/066655 WO2023215863A2 (en) | 2022-05-06 | 2023-05-05 | Rnai agents for modulating snca |
Country Status (1)
Country | Link |
---|---|
WO (1) | WO2023215863A2 (en) |
Citations (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20050244851A1 (en) * | 2004-01-13 | 2005-11-03 | Affymetrix, Inc. | Methods of analysis of alternative splicing in human |
US20140005252A1 (en) * | 2010-11-17 | 2014-01-02 | Isis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
WO2020243292A1 (en) * | 2019-05-28 | 2020-12-03 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing fus expression |
-
2023
- 2023-05-05 WO PCT/US2023/066655 patent/WO2023215863A2/en unknown
Patent Citations (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20050244851A1 (en) * | 2004-01-13 | 2005-11-03 | Affymetrix, Inc. | Methods of analysis of alternative splicing in human |
US20140005252A1 (en) * | 2010-11-17 | 2014-01-02 | Isis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
WO2020243292A1 (en) * | 2019-05-28 | 2020-12-03 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing fus expression |
Also Published As
Publication number | Publication date |
---|---|
WO2023215863A2 (en) | 2023-11-09 |
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